Research into acute myeloid leukaemia (AML)

Researchers around the world are looking at better ways to treat acute myeloid leukaemia (AML). This includes looking at:

  • reducing the number of side effects
  • how treatment affects quality of life Open a glossary item
  • ways to prevent AML from coming back
  • treatment for people whose AML won't go away (refractory) or comes back after treatment (relapsed)

Go to Cancer Research UK's clinical trial database if you are looking for a trial for AML cancer in the UK. Talk to your specialist if there are any trials that you think you might be able to take part in. 

Talk to your specialist if there are any trials that you think you might be able to take part in.

Some of the trials on this page have now stopped recruiting people. It takes time before the results are available. This is because the trial team follow the patients for a period of time and collect and analyse the results. We have included this ongoing research to give examples of the type of research being carried out in AML. 

Research and clinical trials

All cancer treatments must be fully researched before they can be used for everyone. This is so we can be sure that:

  • they work

  • they work better than the treatments already available

  • they are safe

To make sure the research is accurate, each trial has certain entry conditions for who can take part. These are different for each trial.

Hospitals do not take part in every clinical trial. Some trials are only done in a small number of hospitals, or in one area of the country. You may need to travel quite far if you take part in these trials. 

Chemotherapy

Chemotherapy uses anti cancer drugs to destroy cancer cells. You might hear it called cytotoxic Open a glossary item. The drugs circulate throughout the body in the bloodstream. It is the main treatment for AML. There are many trials looking at improving chemotherapy treatment for people with AML. 

Trials are looking into:

  • different combinations of chemotherapy

  • chemotherapy in combination with targeted drugs

  • whether chemotherapy can prevent leukaemia from coming back

  • reducing chemotherapy side effects

  • how chemotherapy affects quality of life

Stem cell transplants

You might have a stem cell or bone marrow transplant as part of your treatment for acute myeloid leukaemia (AML). Most people with AML have a transplant using stem cells or bone marrow from someone else. This is called a donor or allogeneic transplant Open a glossary item.

Half matched (haploidentical) transplants

Doctors can't find a match for about 1 in 3 people who need a transplant. The UK Haplo study is looking at using a half matched transplant in this situation. In a half matched transplant, the donor is at least a 50% match with the person having the stem cell transplant.

Researchers want to find out:

  • how well high dose chemotherapy works with a half matched stem cell transplant

  • how safe it is

  • about side effects

  • how it affects quality of life

Improving stem cell transplants

A hospital in Birmingham is looking at people who are having a stem cell transplant and people who are donors of stem cell transplant. They are looking at blood samples before and after a transplant. They are also taking blood from donors.

The researchers want to look further at these samples to better understand stem cell transplants. 

They hope by having a better understanding of stem cell transplants they can improve the success of treatment and ways to help prevent or lower the side effects.

Another study is looking at new combinations of chemotherapy before a stem cell transplant to improve treatment.

Graft versus host disease (GvHD)

Graft versus host disease (GvHD) is a possible complication of a stem cell transplant from another person. GvHD happens when particular types of white blood cells (T cells) in the donated stem cells or bone marrow attack your own body cells. This is because the donated cells (the graft) see your body cells (the host) as foreign and attack them.

This trial is comparing standard treatment Open a glossary item with 2 newer combinations of treatment to try and see if this helps prevent GvHD.

Giving treatment after a transplant

Researchers want to understand if giving cancer drugs after a stem cell transplant can reduce the risk of their AML coming back. Having further treatment like this is called maintenance treatment. One study is looking at a combination of azacitidine and venetoclax. Azacitidine is a type of chemotherapy and venetoclax is a targeted drug. They are already used in different situations to treat some people with AML. This would be a new way of using these drugs.  

Researchers are looking at a new drug called mocravimod after a stem cell transplant. Mocravimod is a drug that dampens down your immune system Open a glossary item. The helps to reduce the risk of GVHD. The researchers in this study think mocravimod will help reduce the risk of AML coming back.

AML treatment in older people

Treatment for AML varies with age. You have to be very fit to get through some of the intensive treatments. So doctors don't generally use them for older people. The older you are, the less likely you are to be fit enough. The good news is that as we get better at managing side effects, intensive treatments are being used more for older people. 

AML trials are looking into:

  • finding better treatment combinations for older people

  • whether intensive or non intensive treatment works better

  • different chemotherapy drugs

  • whether targeted therapies can help alongside chemotherapy

  • whether new treatments are safe for older people

Targeted cancer drugs

Targeted cancer drugs work by ‘targeting’ those differences that help a cancer cell to survive and grow. There are many different types of targeted drugs with different ways of working. Some seek out and destroy cancer cells. Others help the immune system to attack the cancer. Researchers are looking into:

  • whether certain drugs work best for young people, children or older people

  • different treatment options for people who can't have intensive treatment

  • different drugs for people’s AML that has come back or whose treatment has stopped working

  • new targeted cancer drugs on their own or with other cancer drugs to treat AML

  • whether adding a targeted cancer drug can lower the risk of the AML coming back

Immunotherapies

Immunotherapies can boost the body's own immune system to fight off or kill cancer cells. Researchers in one trial are creating and testing a personal vaccine. This vaccine is made by altering your own leukaemia cells and injecting them back to trigger their immune system. 

CAR T-cell therapy is another treatment that uses your own immune system to treat leukaemia. Doctors remove a type of white blood cells, called T cells. These cells are very good at helping fight infection, but they aren't so good at telling the difference between a normal cell and a cancer cell. Doctors then change the T cells in the laboratory and give them back to you through an infusion. The aim is that the altered T cell recognise and attack any leukaemia cells.

This treatment is used for children with a type of acute lymphoblastic leukaemia. CAR T-cell therapy is now in trial in different situations for children and adults with AML. For example, their disease might not have gone away following treatment or come back after treatment. Doctors want to see if this treatment can put the AML into remission Open a glossary item.

COVID-19 vaccinations and the immune system

Researchers want to find out how well the immune system of people with cancer and chronic illnesses respond to the COVID-19 vaccines.

  • Cancer Research UK Trials database
    Accessed November 2023

  • ClinicalTrials.gov
    Accessed November 2023

  • Acute myeloid leukaemia in adult patients: ESMO Clinical Practice Guidelines for diagnosis, treatment and follow-up
    M Heuser and others
    Annals of Oncology, March 2020. Volume 31, Issue 6, Pages 697 to 712

  • Management of older patients with frailty and acute myeloid leukaemia: A British Society for Haematology good practice paper
    M Dennis and others
    British Journal of Haematology, October 2022. Volume 199, Issue 2, Pages 205 to 221

  • Diagnosis and management of AML in adults: 2022 recommendations from an international expert panel on behalf of the ELN
    H Dohner and others
    Blood, September 2022. Volume 140, Issue 12, Pages 1345 and 1377

  • The information on this page is based on literature searches and specialist checking. We used many references and there are too many to list here. Please contact patientinformation@cancer.org.uk with details of the particular issue you are interested in if you need additional references for this information.

Last reviewed: 
09 Feb 2024
Next review due: 
09 Feb 2027

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